scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution

Background: Adeno-associated virus (AAV)-mediated gene therapies are rapidly advancing to the clinic, and AAV engineering has resulted in vectors with increased ability to deliver therapeutic genes. Although the choice of vector is critical, quantitative comparison of AAVs, especially in large anima...

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Autori principali:	Bilge E Öztürk, Molly E Johnson, Michael Kleyman, Serhan Turunç, Jing He, Sara Jabalameli, Zhouhuan Xi, Meike Visel, Valérie L Dufour, Simone Iwabe, Luis Felipe L Pompeo Marinho, Gustavo D Aguirre, José-Alain Sahel, David V Schaffer, Andreas R Pfenning, John G Flannery, William A Beltran, William R Stauffer, Leah C Byrne
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	eLife Sciences Publications Ltd 2021-10-01
Serie:	eLife
Soggetti:	macaca fascicularis callithrix jacchus scRNA-Seq retinal degeneration adeno-associated virus
Accesso online:	https://elifesciences.org/articles/64175
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